DTX 301

Drug Profile

DTX 301

Alternative Names: DTX 301; Ornithine-transcarbamylase-gene-therapy-Dimension-Therapeutics; OTC-gene-therapy-Dimension-Therapeutics; scAAV8OTC

Latest Information Update: 27 Jul 2018

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At a glance

  • Originator Dimension Therapeutics
  • Class Gene therapies
  • Mechanism of Action Gene transference; Ornithine carbamoyltransferase-expression-stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Inborn urea cycle disorders
  • New Molecular Entity No

Highest Development Phases

  • Phase II Inborn urea cycle disorders

Most Recent Events

  • 18 Jun 2018 Phase-II clinical trials in Inborn urea cycle disorders in Spain (IV) (EudraCT2018-000156-18)
  • 07 Jan 2018 Interim efficacy and adverse events data from a phase I/II trial in Inborn urea cycle disorders released by Ultragenyx Pharmaceuticals
  • 07 Nov 2017 Dimension Therapeutics has been acquired by Ultragenyx Pharmaceutical
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